Top Regulatory News Stories – Week Ending May 31, 2019

AveXis/Novartis received FDA approval for Zolgensma, the gene therapy for children younger than two years old who have been diagnosed with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The therapy will be available to patients for a cost of $2.125 million, or $425,000 per year for 5 years.

Novocure said the FDA approved its NovoTTF-100L System in combination with pemetrexed plus platinum-based chemotherapy as a first-line treatment of unresectable, locally advanced or metastatic, malignant pleural mesothelioma. This marks the first FDA approval for malignant pleural mesothelioma (MPM) in 15 years.

The FDA has approved Incyte Corporation’s Jakafi for treatment of steroid-refractory acute graft-versus-host disease in adult and pediatric patients 12 years and older.

The FDA approved Novartis’s Piqray for breast cancer. This is the first NDA approved under the Real-Time Oncology Review pilot program (RTOR):

Cadent Therapeutics announced that the FDA has granted Orphan Drug Designation to CAD-1883, an investigational treatment for spinocerebellar ataxia (SCA), a genetic disorder characterized by progressive loss of coordination, slurred speech, difficulty controlling eye movements and cognitive dysfunction.

In other news…

Aegerion Pharmaceuticals has entered into a plan funding agreement and a restructuring support agreement that will result in Aegerion selling 100% of its reorganized stock to, and becoming a wholly-owned subsidiary of, Dublin-based Amryt Pharma Plc  

AbbVie noted that depatuxizumab mafodotin (or depatux-​m; previously known as ABT-​414) has been failing to spark a survival benefit in EGFR-​amplified cases of glioblastoma in a Phase III study, causing the independent monitors to call it off for the antibody-​drug conjugate.

Dynavax said it needed to focus all its resources on its 2017-​approved Heplisav-​B and its broader vaccine business, so it is exploring strategic alternatives for its immuno-​oncology programs and cutting jobs.

Stoke Therapeutics has set a preliminary $86 million target for its IPO to help bring its Dravel Syndrome drug into the clinic:

Eli Lilly announced it has inked a license agreement with Centrexion Therapeutics to acquire the exclusive global rights to CNTX-0290. The drug is a small molecule somatostatin receptor type 4 (SSTR4) agonist being evaluated in Phase I as a possible non-opioid therapy for chronic pain.

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